Table 1.
Summary of TEAEs (safety population)
| Double-blind, SAD HVs | Open-label | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| Latozinemab dose level | aFTD-GRN 60 mg/kg (N = 6) | ||||||||
| Pooled placebo (n = 12) | 2 mg/kg (n = 7) | 6 mg/kg (n = 6) | 15 mg/kg (n = 6) | 30 mg/kg (n = 6) | 60 mg/kg (n = 13) | Total AL001 (n = 38) | Total (N = 50) | ||
| Any TEAE, n (%) [E] | 8 (66.7) | 2 (28.6) | 5 (83.3) | 4 (66.7) | 5 (83.3) | 8 (61.5) | 24 (63.2) | 32 (64.0) [70] | 4 (66.7) |
| Any treatment-related TEAE, n (%) [E] | 1 (8.3)a | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (16.7)a | 0 (0.0) | 1 (2.6) | 2 (4.0) | 1 (16.7)b |
| Severity of TEAEs, n (%) [E] | |||||||||
| Mild | 0 (0.0) | 0 (0.0) | 2 (33.3) | 1 (16.7) | 0 (0.0) | 3 (23.1) | 6 (15.8) | 6 (12.0) | 4 (66.7) |
| Moderate | 7 (58.3) | 2 (28.6) | 3 (50.0) | 3 (50.0) | 5 (83.3) | 4 (30.8) | 17 (44.7) | 24 (48.0) | 0 (0.0) |
| Severe | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Life-threatening | 1 (8.3) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (7.7) | 1 (2.6) | 2 (4.0) | 0 (0.0) |
| Death | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Severity of treatment-related TEAEs, n (%) [E] | |||||||||
| Mild | 1 (8.3) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (16.7) | 0 (0.0) | 1 (2.6) | 2 (4.0) | 1 (16.7) |
| Moderate | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Severe | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Life-threatening | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Death | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Any SAE, n (%) [E] | 1 (8.3)c | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (7.7)c | 1 (2.6) | 2 (4.0) | 0 (0.0) |
| Any treatment-related SAE, n (%) [E] | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Any TEAE leading to discontinuation, n (%) [E] | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Any DLAE, n (%) [E] | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | NA |
aFTD-GRN, asymptomatic carrier of GRN mutations causative of frontotemporal dementia; DLAE, dose-limiting adverse event; HV, healthy volunteer; SAD, single ascending dose; TEAE, treatment-emergent adverse event; SAE, serious adverse event
a Post–lumbar puncture syndrome
b Myalgia, lipase increased, and tachycardia
c Rhabdomyolysis